FDA hints relaxed rules are coming for rare disease gene therapy

What will the Trump Administration mean for gene therapy? Researchers, advocates and executives got a glimpse of the answer Thursday, as the Food and Drug Administration convened a “Cell and Gene Therapy Roundtable.”

It was an unusual event. Multiple panelists told STAT on Wednesday they didn’t know what the agenda or goal of the meeting was. There was little debate. No details were hashed out, no specific promises made.

Guests from across academia, advocacy and industry were told to each give a short statement on what the FDA “ought to know” about cell and gene therapy. That took two hours. Then Health Secretary Robert F. Kennedy Jr., National Institutes of Health chief Jay Bhattachrya and Center for Medicaid Services chief Mehmet Oz came in and gave statements. By the time they were d